NL-OMON53946招募中2 期
A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II - DNLI-E-0007
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •1. Participants aged >=2 to <6 years (Cohort A) or >=6 to <17 years (Cohort
- •2. Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS
- •3. For non-run-in Cohort A and Cohort B only: Be on maintenance enzyme
- •replacement therapy (ERT) and have tolerated a minimum of 4 months (ie, 16
- •weeks) of idursulfase therapy during the period immediately prior to screening.
排除标准
- •1. Have a documented pathogenic or likely pathogenic variants that are known to
- •cause developmental delay or decline, cognitive dysfunction, seizures, or other
- •significant CNS disorders
- •2. Previously received an IDS gene therapy or stem cell therapy
- •3. Received any CNS-targeted MPS ERT within 6 months prior to screening
- •4. Have a contraindication for lumbar punctures and/or magnetic resonance
- •imaging (MRIs)
- •5. Participated in any other investigational drug study or used an
- •investigational drug within 60 days prior to screening or intend to receive
- •another investigational drug during the study
研究者
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